PoA = probability-of-approval band (directional only; not FDA guidance)
THIS ISSUE COVERS
The Return of Volume: Liquidity desks return Monday (Jan 5); expect volatility as the holiday “Dead Zone” ends.
The Weekend Warrior: $ATRA ( ▼ 1.77% ) (Tab-cel) faces a rare Saturday PDUFA (Jan 10).
The Mid-Month Cluster: A tight stack of binaries for $TVTX ( ▲ 3.43% ) and $FBIO ( ▲ 10.11% ) next week.
January Calendar: Updated PoA scores for $AQST ( ▼ 0.16% ) T and $PHAR ( ▼ 3.0% ) .
THE WEEK THAT WAS (Recap)
The final two PDUFAs of 2025 resolved during the lowest-liquidity window of the year, with both FDA decisions posting on December 31.
$CORT ( ▲ 9.77% ) — Relacorilant (Hypercortisolism)
PDUFA Date: Dec 30 (Tuesday)
Outcome: Complete Response Letter (CRL) issued Dec 31
Context: The FDA acknowledged that the pivotal GRACE trial met its primary endpoint and that GRADIENT provided supportive confirmatory evidence. However, the agency concluded that the application did not yet provide sufficient evidence of effectiveness to support approval. No approval was granted. Corcept plans to meet with the FDA to discuss next steps.
Market Takeaway: While negative, the CRL narrows the dispute to evidentiary sufficiency rather than trial failure, preserving a defined regulatory path forward.
PDUFA Date: Dec 31 (Wednesday)
Outcome: Approval declined (second rejection in 2025)
Context: The FDA declined approval for ONS-5010/Lytenava, citing insufficient evidence of effectiveness. The agency noted that a key trial did not sufficiently match the comparator, undermining the efficacy case. Additional confirmatory efficacy data were recommended.
Market Takeaway: The decision reinforces the FDA’s high evidentiary bar for ophthalmology assets competing against entrenched standards of care.
LESSONS LEARNED: PoA Calibration from the Year-End Tests
The Dec 31 outcomes stress-tested the PoA framework previewed in the Dec 26 issue under thin liquidity and late-cycle ambiguity.
1) Endpoint Success ≠ Approval Certainty
$CORT ( ▲ 9.77% ) confirms that meeting primary endpoints does not guarantee approval when FDA views the totality of evidence as insufficient.
Calibration: A ~70% PoA appropriately prices meaningful CRL risk in chronic and endocrine indications.
2) Comparator Alignment Is a Hard Gate
$OTLK ( ▼ 58.23% ) underscores that efficacy comparability, especially in wet AMD, is non-negotiable.
Calibration: Sub-40% PoA remains appropriate for efficacy-repair resubmissions with comparator mismatch.
3) Holiday Timing Magnifies Tape Noise, Not Regulatory Leniency
Both decisions posted during the holiday trough. Price reactions were exaggerated; standards were not.
Calibration: PoA should not be “holiday-adjusted.”
Bottom line: The Dec 26 framework held; January sharpens the edges.
MARKET OUTLOOK: The Lights Turn On (Jan 5)
We are now exiting the holiday liquidity vacuum.
As flagged last week, spreads were wide and depth was shallow through Jan 2. Monday, Jan 5 marks the effective return of institutional desks.
What to expect:
Catch-up trades on news released between Dec 26 and Jan 2
Cleaner price discovery as volume normalizes
Renewed focus on near-term binaries rather than placeholders
Names highlighted in last week’s Data Week (Jan 6–10) preview should be monitored closely as liquidity returns.
THE JANUARY BINARY STACK (Jan 10 – Jan 14)
$ATRA ( ▼ 1.77% ) — Tab-cel (EBV+ PTLD)
Date: Jan 10 (Saturday) | PDUFA
PoA: 77 (High)
The Setup: Priority Review BLA for EBV-positive post-transplant lymphoproliferative disease. If approved, Tab-cel would become the first U.S.-approved therapy for this indication.
Our View: Saturday PDUFAs are operationally tricky; announcements often post Friday after the close or Monday pre-market. Pivotal data showed 50.7% ORR with 18.4 months median overall survival, providing a strong clinical floor. Approval would also trigger a reported $40M milestone payment from partner Pierre Fabre, underscoring the commercial significance of a positive decision.
$TVTX ( ▲ 3.43% ) — Filspari (FSGS, Full Approval)
Date: Jan 13 (Tuesday) | PDUFA
PoA: 70 (High)
The Setup: sNDA seeking conversion from accelerated to traditional approval in focal segmental glomerulosclerosis.
Our View: Supported by Phase 3 DUPLEX and Phase 2 DUET data. Approval would cement Filspari as the first fully FDA-approved therapy for FSGS.
$FBIO ( ▲ 10.11% ) — CUTX-101 (Menkes Disease)
Date: Jan 14 (Wednesday) | PDUFA
PoA: 80 (High)
The Setup: Pediatric Menkes disease therapy under review following a Class 1 NDA resubmission.
Our View: Elevated PoA reflects the administrative nature of the resubmission in an ultra-rare pediatric setting.
JANUARY PDUFA CALENDAR
Ticker | Date | Event | PoA | Summary |
|---|---|---|---|---|
Jan 10 | Tab-cel (EBV+ PTLD) | 77 | First-in-class allogeneic T-cell therapy | |
Jan 13 | Filspari (FSGS) | 70 | Full approval decision based on DUPLEX | |
Jan 14 | CUTX-101 (Menkes) | 80 | Class 1 resubmission | |
Jan 31* | Anaphylm (Epi Film) | 80 | First oral epinephrine film | |
Jan 31* | Leniolisib (Pediatric APDS) | 85 | Priority Review pediatric sNDA |
* Jan 31 falls on a Saturday; FDA action may post Friday after the close or Monday pre-market. Note: $PHAR ( ▼ 3.0% ) represents a pediatric sNDA expansion of an already-approved drug, which historically carries lower volatility than first-in-class approvals such as $AQST ( ▼ 0.16% ) .
DISCLAIMER
This newsletter is for informational purposes only. Biotech securities involve substantial risk, including total loss of capital. Always conduct your own due diligence.
Related Research
For readers interested in deeper regulatory precedent analysis, we’ve published a standalone PDUFA Risk Dossier examining how the FDA has historically resolved CMC-driven CRLs in gene therapy, using Rocket Pharmaceuticals ($RCKT) as a case study. [Link]